{ "api_version": "1", "request": { "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=GM1+Gangliosidoses", "query": { "condition": "GM1 Gangliosidoses" }, "page_size": 10 }, "pagination": { "page": 1, "page_size": 10, "total_count": 20, "total_pages": 2, "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=GM1+Gangliosidoses&page=2&page_size=10", "previous_page_url": null }, "source": "remote", "last_synced_at": "2026-05-21T23:40:47.808Z", "attribution": "Data derived from public ClinicalTrials.gov records. The official record at https://clinicaltrials.gov/ remains the source of truth for current availability, contacts, and full study details.", "notes": [ "This endpoint exposes only public summary fields — no participant contact emails, phone numbers, or scraped investigator contact details.", "For contact information and full protocol detail, follow each trial's `official_url` to the ClinicalTrials.gov record.", "This API is a navigational aid. It does not provide medical advice, eligibility determinations, or compensation guarantees." ], "trials": [ { "nct_id": "NCT06539169", "title": "FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases", "overall_status": "RECRUITING", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Alpha-Thalassemia", "Beta-Thalassemia", "Amyloidosis", "Amyotrophic Lateral Sclerosis", "Creutzfeld-Jakob Disease", "Cystic Fibrosis", "Duchenne Muscular Dystrophy", "Early-Onset Alzheimer Disease", "Ehlers-Danlos Syndrome", "Huntington Disease", "Gaucher Disease", "GM1 Gangliosidosis", "Myasthenia Gravis", "Pompe Disease", "Sickle Cell Disease", "Transthyretin Amyloid Cardiomyopathy", "Rare Diseases" ], "interventions": [], "intervention_types": [], "sponsor": "xCures", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": null, "sex": "ALL", "summary": "Not listed" }, "enrollment_count": 1000, "start_date": "2024-06-10", "completion_date": "2026-06-10", "has_results": false, "last_update_posted_date": "2024-11-14", "last_synced_at": "2026-05-21T23:40:47.808Z", "location_count": 1, "location_summary": "Los Altos, California", "locations": [ { "city": "Los Altos", "state": "California" } ], "official_url": "https://clinicaltrials.gov/study/NCT06539169" }, { "nct_id": "NCT04041102", "title": "Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients", "overall_status": "COMPLETED", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "GM1 Gangliosidosis" ], "interventions": [], "intervention_types": [], "sponsor": "University of Pennsylvania", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": null, "sex": "ALL", "summary": "Not listed" }, "enrollment_count": 31, "start_date": "2020-06-12", "completion_date": "2024-05-01", "has_results": false, "last_update_posted_date": "2025-10-24", "last_synced_at": "2026-05-21T23:40:47.808Z", "location_count": 2, "location_summary": "Oakland, California • Philadelphia, Pennsylvania", "locations": [ { "city": "Oakland", "state": "California" }, { "city": "Philadelphia", "state": "Pennsylvania" } ], "official_url": "https://clinicaltrials.gov/study/NCT04041102" }, { "nct_id": "NCT00029965", "title": "Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders", "overall_status": "RECRUITING", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Neurological Regression", "Myoclonus", "Cherry Red Spot", "Brain Atrophy" ], "interventions": [], "intervention_types": [], "sponsor": "National Human Genome Research Institute (NHGRI)", "sponsor_class": "NIH", "healthy_volunteers": false, "eligibility": { "minimum_age": "1 Day", "maximum_age": "100 Years", "sex": "ALL", "summary": "1 Day to 100 Years" }, "enrollment_count": 200, "start_date": "2002-02-06", "completion_date": null, "has_results": false, "last_update_posted_date": "2026-04-29", "last_synced_at": "2026-05-21T23:40:47.808Z", "location_count": 1, "location_summary": "Bethesda, Maryland", "locations": [ { "city": "Bethesda", "state": "Maryland" } ], "official_url": "https://clinicaltrials.gov/study/NCT00029965" }, { "nct_id": "NCT07054515", "title": "A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis", "overall_status": "RECRUITING", "study_type": "INTERVENTIONAL", "phases": [ "PHASE3" ], "conditions": [ "Niemann-Pick Type C Disease", "GM1 Gangliosidosis", "GM2 Gangliosidosis" ], "interventions": [ { "name": "AZ-3102", "type": "DRUG" }, { "name": "Placebo", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Azafaros A.G.", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": "4 Years", "maximum_age": null, "sex": "ALL", "summary": "4 Years and older" }, "enrollment_count": 147, "start_date": "2025-06-30", "completion_date": "2027-11-04", "has_results": false, "last_update_posted_date": "2026-05-05", "last_synced_at": "2026-05-21T23:40:47.808Z", "location_count": 5, "location_summary": "Oakland, California • Minneapolis, Minnesota • Rochester, Minnesota + 2 more", "locations": [ { "city": "Oakland", "state": "California" }, { "city": "Minneapolis", "state": "Minnesota" }, { "city": "Rochester", "state": "Minnesota" }, { "city": "Dallas", "state": "Texas" }, { "city": "Fairfax", "state": "Virginia" } ], "official_url": "https://clinicaltrials.gov/study/NCT07054515" }, { "nct_id": "NCT03952637", "title": "A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis", "overall_status": "RECRUITING", "study_type": "INTERVENTIONAL", "phases": [ "PHASE1", "PHASE2" ], "conditions": [ "Lysosomal Diseases", "Gangliosidosis", "GM1" ], "interventions": [ { "name": "AAV9-GLB1", "type": "BIOLOGICAL" }, { "name": "Abdominal ultrasound", "type": "PROCEDURE" }, { "name": "Rituximab", "type": "DRUG" }, { "name": "Sirolimus", "type": "DRUG" }, { "name": "Methylprednisolone", "type": "DRUG" }, { "name": "Prednisone", "type": "DRUG" }, { "name": "Audiology assessment with ABR", "type": "DIAGNOSTIC_TEST" }, { "name": "Bone density scan (DEXA)", "type": "DIAGNOSTIC_TEST" }, { "name": "Electrocardiogram (EKG)", "type": "DIAGNOSTIC_TEST" }, { "name": "Echocardiogram", "type": "DIAGNOSTIC_TEST" }, { "name": "Electroencephalogram (EEG) awake and extended overnight", "type": "OTHER" }, { "name": "Laboratory tests", "type": "DIAGNOSTIC_TEST" }, { "name": "Lumbar puncture", "type": "PROCEDURE" }, { "name": "Brain MRI/MRS/fMRI", "type": "PROCEDURE" }, { "name": "Neurocognitive testing", "type": "BEHAVIORAL" }, { "name": "Neurology exam", "type": "OTHER" }, { "name": "PICC or other Central line placement", "type": "BEHAVIORAL" }, { "name": "Skeletal survey", "type": "PROCEDURE" }, { "name": "Skin biopsy", "type": "PROCEDURE" }, { "name": "Speech and modified barium swallow study", "type": "PROCEDURE" }, { "name": "Ophthalmology exam", "type": "PROCEDURE" } ], "intervention_types": [ "BIOLOGICAL", "PROCEDURE", "DRUG", "DIAGNOSTIC_TEST", "OTHER", "BEHAVIORAL" ], "sponsor": "National Human Genome Research Institute (NHGRI)", "sponsor_class": "NIH", "healthy_volunteers": false, "eligibility": { "minimum_age": "6 Months", "maximum_age": "12 Years", "sex": "ALL", "summary": "6 Months to 12 Years" }, "enrollment_count": 54, "start_date": "2019-08-19", "completion_date": "2028-01-01", "has_results": false, "last_update_posted_date": "2025-12-11", "last_synced_at": "2026-05-21T23:40:47.808Z", "location_count": 1, "location_summary": "Bethesda, Maryland", "locations": [ { "city": "Bethesda", "state": "Maryland" } ], "official_url": "https://clinicaltrials.gov/study/NCT03952637" }, { "nct_id": "NCT03333200", "title": "Longitudinal Study of Neurodegenerative Disorders", "overall_status": "RECRUITING", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "MLD", "Krabbe Disease", "ALD", "MPS I", "MPS II", "MPS III", "Vanishing White Matter Disease", "GM3 Gangliosidosis", "PKAN", "Tay-Sachs Disease", "NP Deficiency", "Osteopetrosis", "Alpha-Mannosidosis", "Sandhoff Disease", "Niemann-Pick Diseases", "MPS IV", "Gaucher Disease", "GAN", "GM1 Gangliosidoses", "Morquio Disease", "S-Adenosylhomocysteine Hydrolase Deficiency", "Batten Disease", "Pelizaeus-Merzbacher Disease", "Leukodystrophy", "Lysosomal Storage Diseases", "Purine Nucleoside Phosphorylase Deficiency", "Multiple Sulfatase Deficiency Disease" ], "interventions": [ { "name": "Palliative Care", "type": "OTHER" }, { "name": "Hematopoetic Stem Cell Transplantation", "type": "BIOLOGICAL" } ], "intervention_types": [ "OTHER", "BIOLOGICAL" ], "sponsor": "University of Pittsburgh", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": null, "sex": "ALL", "summary": "Not listed" }, "enrollment_count": 1500, "start_date": "2012-01-11", "completion_date": "2035-01", "has_results": false, "last_update_posted_date": "2026-02-09", "last_synced_at": "2026-05-21T23:40:47.808Z", "location_count": 1, "location_summary": "Pittsburgh, Pennsylvania", "locations": [ { "city": "Pittsburgh", "state": "Pennsylvania" } ], "official_url": "https://clinicaltrials.gov/study/NCT03333200" }, { "nct_id": "NCT01626092", "title": "Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "NA" ], "conditions": [ "Lysosomal Storage Disease", "Peroxisomal Disorder" ], "interventions": [ { "name": "Campath-1H", "type": "DRUG" }, { "name": "Clofarabine", "type": "DRUG" }, { "name": "Melphalan", "type": "DRUG" }, { "name": "Total Body Irradiation with Marrow Boosting", "type": "RADIATION" }, { "name": "Hematopoietic stem cell transplantation", "type": "BIOLOGICAL" }, { "name": "Cyclosporine A", "type": "DRUG" }, { "name": "Mycophenolate mofetil", "type": "DRUG" } ], "intervention_types": [ "DRUG", "RADIATION", "BIOLOGICAL" ], "sponsor": "Masonic Cancer Center, University of Minnesota", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": "55 Years", "sex": "ALL", "summary": "Up to 55 Years" }, "enrollment_count": 3, "start_date": "2012-07-11", "completion_date": "2013-11", "has_results": true, "last_update_posted_date": "2017-12-05", "last_synced_at": "2026-05-21T23:40:47.808Z", "location_count": 1, "location_summary": "Minneapolis, Minnesota", "locations": [ { "city": "Minneapolis", "state": "Minnesota" } ], "official_url": "https://clinicaltrials.gov/study/NCT01626092" }, { "nct_id": "NCT07082725", "title": "A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC)", "overall_status": "RECRUITING", "study_type": "INTERVENTIONAL", "phases": [ "PHASE3" ], "conditions": [ "Niemann-Pick Type C Disease" ], "interventions": [ { "name": "Nizubaglustat", "type": "DRUG" }, { "name": "Placebo", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Azafaros A.G.", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": "4 Years", "maximum_age": null, "sex": "ALL", "summary": "4 Years and older" }, "enrollment_count": 72, "start_date": "2025-06-30", "completion_date": "2027-11-04", "has_results": false, "last_update_posted_date": "2026-05-05", "last_synced_at": "2026-05-21T23:40:47.808Z", "location_count": 5, "location_summary": "Oakland, California • Minneapolis, Minnesota • Rochester, Minnesota + 2 more", "locations": [ { "city": "Oakland", "state": "California" }, { "city": "Minneapolis", "state": "Minnesota" }, { "city": "Rochester", "state": "Minnesota" }, { "city": "Dallas", "state": "Texas" }, { "city": "Fairfax", "state": "Virginia" } ], "official_url": "https://clinicaltrials.gov/study/NCT07082725" }, { "nct_id": "NCT02030015", "title": "Synergistic Enteral Regimen for Treatment of the Gangliosidoses", "overall_status": "TERMINATED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE4" ], "conditions": [ "GM1 Gangliosidoses", "GM2 Gangliosidoses", "Tay-Sachs Disease", "Sandhoff Disease" ], "interventions": [ { "name": "miglustat", "type": "DRUG" }, { "name": "Ketogenic Diet", "type": "OTHER" } ], "intervention_types": [ "DRUG", "OTHER" ], "sponsor": "University of Minnesota", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": "204 Months", "sex": "ALL", "summary": "Up to 204 Months" }, "enrollment_count": 16, "start_date": "2015-12-22", "completion_date": "2019-07-31", "has_results": true, "last_update_posted_date": "2021-04-14", "last_synced_at": "2026-05-21T23:40:47.808Z", "location_count": 1, "location_summary": "Minneapolis, Minnesota", "locations": [ { "city": "Minneapolis", "state": "Minnesota" } ], "official_url": "https://clinicaltrials.gov/study/NCT02030015" }, { "nct_id": "NCT00654433", "title": "ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases", "overall_status": "TERMINATED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE3" ], "conditions": [ "Inherited Metabolic Diseases", "Lysosomal Storage Disorders", "Peroxisomal Storage Diseases", "Inborn Errors of Metabolism", "Mucopolysaccharidosis" ], "interventions": [ { "name": "ALD-101", "type": "BIOLOGICAL" } ], "intervention_types": [ "BIOLOGICAL" ], "sponsor": "Aldagen", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": "16 Years", "sex": "ALL", "summary": "Up to 16 Years" }, "enrollment_count": 40, "start_date": "2008-03", "completion_date": "2011-11", "has_results": false, "last_update_posted_date": "2014-07-08", "last_synced_at": "2026-05-21T23:40:47.808Z", "location_count": 3, "location_summary": "Los Angeles, California • New York, New York • Durham, North Carolina", "locations": [ { "city": "Los Angeles", "state": "California" }, { "city": "New York", "state": "New York" }, { "city": "Durham", "state": "North Carolina" } ], "official_url": "https://clinicaltrials.gov/study/NCT00654433" } ] }